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ClinicalTrials.gov public records Last synced Sep 6, 2026, 5:33 AM EDT

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Showing 1–24 of 32 matching trials from the live ClinicalTrials.gov search.
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Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation

NCT07588581
View directory record Official record
Conditions
Childhood-Onset Neurodegeneration With Brain Atrophy (CONDBA)
Interventions
nL-UBTF-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced Sep 6, 2026, 5:33 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation

NCT07084311
View directory record Official record
Conditions
Dentatorubral-Pallidoluysian Atrophy
Interventions
nL-ATN1-002
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
29 Years to 29 Years · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Honolulu, Hawaii
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 6, 2026, 5:33 AM EDT
Enrolling by invitation Early Phase 1 Interventional

Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1

NCT06565572
View directory record Official record
Conditions
Posterior Column Ataxia With Retinitis Pigmentosa
Interventions
nL-FLVC-001
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced Sep 6, 2026, 5:33 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A

NCT06314490
View directory record Official record
Conditions
Genetic Disease
Interventions
nL-SCN2A-002
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Mar 31, 2025 · Synced Sep 6, 2026, 5:33 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for a Single Participant With MAPK8IP3 Neurodevelopmental Disorder With or Without Variable Brain Abnormalities (NEDBA)

NCT07197294
View directory record Official record
Conditions
Neurodevelopmental Disorder With or Without Variable Brain Abnormalities
Interventions
nL-MAPK8-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
5 Years to 5 Years · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 28, 2025 · Synced Sep 6, 2026, 5:33 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for a Single Participant With PRPH2 Mutation Associated With Retinal Dystrophy

NCT07177196
View directory record Official record
Conditions
Retinal Dystrophy
Interventions
nL-PRPH2-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Sep 15, 2025 · Synced Sep 6, 2026, 5:33 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation

NCT07221760
View directory record Official record
Conditions
Dentatorubral-Pallidoluysian Atrophy
Interventions
nL-ATN1-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
22 Years and older · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Apr 6, 2026 · Synced Sep 6, 2026, 5:33 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS

NCT07743268
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis (ALS)
Interventions
nL-TARDB-006
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
Not listed
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
2
States / cities
St Louis, Missouri • New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 2, 2026 · Synced Sep 6, 2026, 5:33 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS

NCT07095686
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
nL-CHCHD-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 6, 2026, 5:33 AM EDT
Completed Phase 1 Interventional

Trial of Combination Therapy to Treat COVID-19 Infection

NCT04482686
View directory record Official record
Conditions
COVID, Covid-19, Corona Virus Infection, Coronavirus Infection, Coronavirus-19, SARS-CoV2, SARS-CoV Infection
Interventions
Ivermectin, Doxycycline Hcl, Zinc, Vitamin D3, Vitamin C
Drug · Dietary Supplement
Lead sponsor
ProgenaBiome
Other
Eligibility
18 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Ventura, California
Source: ClinicalTrials.gov public record
Updated Sep 23, 2024 · Synced Sep 6, 2026, 5:33 AM EDT
Completed Phase 2 Interventional Results available

Ruxolitinib Cream in the Treatment of Cutaneous Necrobiosis Lipoidica

NCT04492618
View directory record Official record
Conditions
Necrobiosis Lipoidica
Interventions
Ruxolitinib
Drug
Lead sponsor
Aaron R. Mangold
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Scottsdale, Arizona
Source: ClinicalTrials.gov public record
Updated Feb 7, 2023 · Synced Sep 6, 2026, 5:33 AM EDT
Completed No phase listed Observational

Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis

NCT01144507
View directory record Official record
Conditions
Cystic Fibrosis, Pancreatic Insufficiency
Interventions
Abdominal Ultrasound, Sample collection procedures
Procedure · Other
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
3 Years to 12 Years
Enrollment
774 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2023
U.S. locations
10
States / cities
Aurora, Colorado • Atlanta, Georgia • Chicago, Illinois + 7 more
Source: ClinicalTrials.gov public record
Updated Apr 10, 2025 · Synced Sep 6, 2026, 5:33 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD)

NCT06816498
View directory record Official record
Conditions
Autosomal Dominant Leukodystrophy
Interventions
nL-LMNB1-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
51 Years to 51 Years · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 6, 2026, 5:33 AM EDT
Recruiting No phase listed Observational

Impact of Highly Effective Modulator Therapy on the Cystic Fibrosis Microbiome

NCT05982795
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Nasal endoscopy with microbiome swab and mucus collection through filter paper and sponge, Olfactory Function Testing, Survey Completion
Other
Lead sponsor
Dartmouth-Hitchcock Medical Center
Other
Eligibility
18 Years to 99 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Lebanon, New Hampshire
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Sep 6, 2026, 5:33 AM EDT
Completed No phase listed Observational

Sleep Disordered Breathing and Alzheimer's Disease Biomarkers in Normal Aging and Mild Cognitive Impairment

NCT03912571
View directory record Official record
Conditions
Sleep Disorder
Interventions
Structural 3T MRI, MRI Based Perfusion Imaging, VR-CO2 MRI Scans
Diagnostic Test
Lead sponsor
NYU Langone Health
Other
Eligibility
55 Years to 90 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 4, 2021 · Synced Sep 6, 2026, 5:33 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576)

NCT07423494
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
nL-CHCHD-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 19, 2026 · Synced Sep 6, 2026, 5:33 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS

NCT06977451
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
nL-CHCHD-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
63 Years to 63 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 6, 2026, 5:33 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy

NCT06369974
View directory record Official record
Conditions
Genetic Disease
Interventions
Antisense oligonucleotide treatment (ASO)
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
4 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 1, 2026 · Synced Sep 6, 2026, 5:33 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy

NCT07222371
View directory record Official record
Conditions
Genetic Disease, TUBB4A-Related Leukodystrophy
Interventions
nL-TUBB4-001
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Oct 28, 2025 · Synced Sep 6, 2026, 5:33 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

NCT06392126
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
nL-CHCHD-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
48 Years and older · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Jacksonville, Florida
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 6, 2026, 5:33 AM EDT
Completed Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS

NCT07095712
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
nL-TARD-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
49 Years to 49 Years · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
2
States / cities
New York, New York • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Sep 6, 2026, 5:33 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)

NCT07226297
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease Type 2D
Interventions
nL-GARS1-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
13 Years and older · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 9, 2025 · Synced Sep 6, 2026, 5:33 AM EDT
Completed No phase listed Observational

PhosphoRus, Proton Imaging and Amyloid BuRdEn (PREPARE) ON AMYLOID BURDEN AND COGNITION

NCT03999879
View directory record Official record
Conditions
Alzheimer Disease
Interventions
Measure of OxPhos upregulation, lactate (measured with 1H-MRSI)
Diagnostic Test
Lead sponsor
NYU Langone Health
Other
Eligibility
70 Years to 85 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 2, 2023 · Synced Sep 6, 2026, 5:33 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available

Study Comparing Prandial Insulin Aspart vs. Technosphere Insulin in Patients With Type 1 Diabetes on Multiple Daily Injections: Investigator-Initiated A Real-life Pilot Study-STAT Study

NCT03143816
View directory record Official record
Conditions
Diabetes Mellitus, Type 1
Interventions
Technosphere insulin
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years to 70 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Feb 21, 2022 · Synced Sep 6, 2026, 5:33 AM EDT